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KEGG. D11559. Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), [6][7] a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. [6] It works by providing a new copy of the SMN gene that produces the SMN protein.
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [1][5][2] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015. [6][7] The most common adverse reactions include reduced platelet and other ...
Novartis Gene Therapies, until 2020 known as AveXis, is a biotechnology company that develops treatments for rare neurological genetic disorders. It was founded in Dallas , Texas , United States in 2012 by John Carbona after reorganizing a company called BioLife Cell Bank founded by David Genecov and John Harkey.
The therapy has a US list price of US$2.2 million. [16] The cost effectiveness threshold of the therapy in the US is estimated to be between $1.35 million and $2.05 million [ 17 ] depending on perspective (healthcare vs limited societal) and assuming the willingness to pay for 1 quality-adjusted life year (QALY) at $100,000–$150,000.
The modified gene therapy strategy of antisense IGF-I RNA (NIH n˚ 1602) [162] using antisense / triple helix anti-IGF-I approach was registered in 2002, by Wiley gene therapy clinical trial - n˚ 635 and 636.
Virotherapy. Virotherapy is a treatment using biotechnology to convert viruses into therapeutic agents by reprogramming viruses to treat diseases. There are three main branches of virotherapy: anti-cancer oncolytic viruses, viral vectors for gene therapy and viral immunotherapy. These branches use three different types of treatment methods ...
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